Researchers from Universitätsklinikum Erlangen, Germany, have used an experimental immunotherapy to give a teenage girl her life back, after she was diagnosed with a deadly autoimmune disease at age 16. Their research was published in the New England Journal of Medicine.

Troubled with severe joint pain and rashes over her body, Thu-Thao V was unable to continue her hobbies and faced life-threatening complications as the systemic lupus erythematosus (SLE) could target any of her organs at any time.  

Thu-Thao V had to take extensive medication to control her symptoms, and doctors tried everything available to prevent them. She was treated with hydroxychloroquine, steroids, and currently available B-cell immunotherapies, but nothing worked and her joint pain continued. 

So, doctors turned to a new type of immunotherapy. Currently in use for certain types of aggressive cancer and leukemia, chimeric antigen receptor T-cell (CAR-T) therapy reprogrammes aberrant immune cells in the patient’s body, allowing them to recognize and destroy tumors. However, B-cells (the target of the therapy) are also heavily implicated in lupus, in which they create antibodies that directly target double-stranded DNA. It is thought that if the researchers can use CAR-T therapy to deplete B-cell numbers, there will be fewer circulating autoantibodies that cause the devastating lupus symptoms. 

"CAR stands for chimeric antigen receptor which is an artificial receptor," explains Professor Dr. Andreas Mackensen, Director of the Department of Medicine 5 – Haematology and Oncology, in a statement. 

"Immune cells, or T cells, from the patient are genetically engineered in the laboratory to add the CAR. The CAR recognises special antigens on the surface of the target cells and destroys them. Cell therapy with CAR-T cells is already being successfully used to treat leukaemia and lymphoma.’ 

In March 2021, Thu-Thao V was treated at Universitätsklinikum Erlangen with CAR-T cells.

Following the therapy, the CAR-T cell numbers rapidly increased and remained circulating in her system. This then resulted in a rapid depletion of B-cells and autoantibodies thought to be the cause of the autoimmune symptoms. Just six months after the treatment, Thu-Thao V is in complete remission and has returned to sports, something that she was unable to do while the joint pain, heart palpitations, and renal issues were present. She no longer requires drugs, and all her symptoms have disappeared. 

This is a massive breakthrough in immunotherapy, presenting an in vivo use of CAR-T cell therapy to put a young girl with a severe autoimmune disease into complete remission for a long period of time after therapy. The researchers now hope to continue this work into a clinical trial on people with autoimmune diseases.